Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: An observational cohort study

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Patterson, Marc C ^a   Mengel, Eugen ^b   Vanier, Marie T ^c   Schwierin, Barbara ^d   Muller, Audrey ^d   Cornelisse, Peter ^d   Pineda, Mercè ^e   Amado Fondo, A ^f   Amraoui, Y ^f   Andria, G ^f   Arellano, M ^f   Audoin, B ^f   Azcona, C ^f   Barr, C ^f   Baruteau, J ^f   Baumgartner, C ^f   Bell, L ^f   Bembi, B ^f   Benneddif, K ^f   Bernard, G ^f   Bobocea, N ^f   Bodzioch, M ^f   Boettcher, T ^f   Bonnan, M ^f   Broue, P ^f   Bruni, A ^f   Caceres, M ^f   Camino, R ^f   Campbell, E ^f   Cances, C ^f   Cannell, P ^f   Cesar, J ^f   Chabrol, B ^f   Chakrapani, A ^f   Colao, R ^f   Collet, A ^f   Corsetti, T ^f   Cousins, A ^f   Covanis, A ^f   Cox, T ^f   Cuisset, J M ^f   Dardis, A ^f   Das, A ^f   Deegan, P ^f   Dengler, T ^f   Deodato, F ^f   Derralynn, H ^f   Di Donato, I ^f   Di Rocco, M ^f   Dinopoulos, A ^f   Pakiela, Domanska ^f   Eckehard, S ^f   Engelen, M ^f   Eyer, D ^f   Fecarotta, S ^f   Federico, A ^f   Filla, A ^f   Fiumara, A ^f   Fonseca, M J ^f   Gabrielli, O ^f   Garcia, T ^f   Garrote, J ^f   Gissen, P ^f   Giugliani, L ^f   Greenberg, C ^f   Heron, B ^f   Hertzberg, C ^f   Higgins, F ^f   Hill, A ^f   Hiwot, T ^f   Hlavata, A ^f   Horbe Blindt A ^f   Howley, E ^f   Hussain, N ^f   Illsinger, S ^f   Imrie, J ^f   Jacklin, E ^f   Jones, S ^f   Jovanovic, A ^f   Kaczmarek, V ^f   Kaphan, E ^f   Kibaek, M ^f   Kleinhans, P ^f   Klunemann K H ^f   Koch, S M ^f   Koegl Wallner, W ^f   Kolnikova, M ^f   Korenke, G C ^f   Korinthenberg, R ^f   Kumari, S ^f   Lachmann, R ^f   Lee Ann, L ^f   Likopoulou, L ^f   Lilienthal, E ^f   Link, B ^f   Lippold, M ^f   Lopez Laso, E ^f   Luecke, T ^f   Lundgren, J ^f   Mackrell, M ^f   Madruga, M ^f   Maletta, R ^f   Malinova, V ^f   Manners, J ^f   Marinei, R ^f   Marquardt, T ^f   Martins, E ^f   Martins, A M ^f   Martins, N ^f   McAlister, L ^f   McCabe, A ^f   McKie, M ^f   McMahon, S ^f   Meehan, M ^f   Meldgaard Lund, A ^f   Mendozah, C ^f   Meyer, A ^f   Mielke, S ^f   Milligan, A ^f   Mir, P ^f   Moisa, M ^f   Mombelli, C ^f   Morris, L ^f   Muller Vom Hagen J ^f   Munoz, B ^f   Murphy, E ^f   Nagarajan, L ^f   Neto, P B ^f   Nevsimalova, S ^f   Nia, S ^f   Nicolai, J ^f   Niemann, D ^f   Niktari, G ^f   O'Callaghan, M D M ^f   Paucar Arce, M ^f   Peers, K ^f   Peintinger, L ^f   Peralta, M ^f   Perez J ^f   Perez Poyato, M ^f   Petrariu, A ^f   Puschmann, A ^f   Raiman, J ^f   Rask, O ^f   Rataj, J ^f   Raymond Gaynor, C ^f   Reichelt, G ^f   Ribeiro, E ^f   Riches, V ^f   Roberts, A ^f   Roelants, J ^f   Rohrbach, M ^f   Rokicki, D ^f   Rolfs, A ^f   Russo, C ^f   Rutsch, F ^f   Saleem, R ^f   Santos, M ^f   Schmutz, P ^f   Schwahn, B ^f   Sedel, F ^f   Semotok, J ^f   Sharma, R ^f   Silska, S ^f   Silva, A ^f   Simmons, L ^f   Sivera, R ^f   Skorpen, J ^f   Sole, G ^f   Souza, C ^f   Stadlober Degwerth, M ^f   Starling, J ^f   Temudo, T ^f   Tomas, M ^f   Tranchant, C ^f   Uziel, G ^f   Valayannopoulous, V ^f   Van Den Hout, H ^f   Van Der Tol, L ^f   Van Spronsen, F ^f   Vellodi, A ^f   Verdu, A ^f   Vilchez, J J ^f   Vinaixa, A ^f   Visser, G ^f   Voelker, J ^f   Waldek, S ^f   Walter, A ^f   Walterfang, M ^f   Wein, U ^f   Widner, H ^f   Wilcke, C ^f   Wildish, L ^f   Wraith, E ^f   Wright, N ^f   Xaidara, A ^f   Yamamoto, M ^f   Zallocco, F ^f   Zielke, S ^f   more..

^a MAYO CLINIC   (United States)

^b JOHANNES GUTENBERG UNIVERSITY   (Germany)

^c INSERM   (France)

^d ACTELION PHARMACEUTICALS LTD   (Switzerland)

^e CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED DE ENFERMEDADES RARAS CIBERER   (Spain)

^f NONE

Author keywords

Miglustat; Niemann Pick disease type C; Registry

Indexed keywords

MIGLUSTAT; 1 DEOXYNOJIRIMYCIN; ENZYME INHIBITOR;

ADOLESCENT; ADULT; AGED; ARTICLE; CHRONIC DIARRHEA; CLINICAL EFFECTIVENESS; COHORT ANALYSIS; DISABILITY; DISEASE COURSE; DISEASE REGISTRY; DRUG EFFICACY; DRUG EXPOSURE; FEMALE; FOLLOW UP; HUMAN; LONGITUDINAL STUDY; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; NEUROPATHY; NIEMANN PICK DISEASE; OBSERVATIONAL STUDY; PATIENT SAFETY; SEIZURE; THROMBOCYTOPENIA; TREMOR; ANALOGS AND DERIVATIVES; CHILD; NIEMANN-PICK DISEASE, TYPE C; PATHOLOGY; PRESCHOOL CHILD; PROSPECTIVE STUDY; TREATMENT OUTCOME; YOUNG ADULT;

1-DEOXYNOJIRIMYCIN; ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; DISEASE PROGRESSION; ENZYME INHIBITORS; FEMALE; HUMANS; MALE; NIEMANN-PICK DISEASE, TYPE C; PROSPECTIVE STUDIES; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84931039342     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0284-z     Document Type: Article

References (22)

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